New drug shows promise in reducing Liver-Damaging protein in rare disease
NCT ID NCT03945292
First seen Nov 04, 2025 · Last updated May 24, 2026 · Updated 26 times
Summary
This study tested an experimental drug called fazirsiran in 40 adults with alpha-1 antitrypsin deficiency, a genetic condition that can cause liver and lung damage. The goal was to see if the drug safely lowers levels of a harmful protein (Z-AAT) in the blood. Participants received multiple doses or a placebo, and researchers measured protein changes and side effects over 16 weeks.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Research Site 1
Charleston, South Carolina, 29425, United States
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Research Site 10
St Louis, Missouri, 63104, United States
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Research Site 11
Sacramento, California, 95817, United States
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Research Site 12
New York, New York, 10032, United States
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Research Site 13
Stanford, California, 94305, United States
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Research Site 14
La Jolla, California, 92037, United States
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Research Site 15
San Francisco, California, 94158, United States
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Research Site 16
Salt Lake City, Utah, 84132, United States
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Research Site 17
Los Angeles, California, 90095, United States
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Research Site 19
Houston, Texas, 77030, United States
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Research Site 2
Gainesville, Florida, 32610, United States
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Research Site 20
Barcelona, 08035, Spain
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Research Site 22
Pavia, 27100, Italy
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Research Site 23
Leiden, 2333 ZA, Netherlands
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Research Site 24
Funchal, 9004-514, Portugal
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Research Site 27
Aachen, 52074, Germany
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Research Site 3
Iowa City, Iowa, 52242, United States
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Research Site 4
Nashville, Tennessee, 37232, United States
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Research Site 5
Birmingham, Alabama, 35233, United States
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Research Site 7
Indianapolis, Indiana, 46202, United States
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Research Site 9
Phoenix, Arizona, 85054, United States
Conditions
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